Acute myeloid leukemia treatment: Navigating your care path
Fact-checked by Acute myeloid leukemia (AML) treatment often involves a combination of several approaches, including chemotherapy, targeted therapies, supportive care, and, for some people, a stem cell transplant.
Because AML often progresses quickly, treatment usually needs to begin soon after a diagnosis is made. The first goal is to achieve remission by eliminating as many leukemia cells as possible. Later stages of treatment aim to destroy leukemia cells that may remain and reduce the risk of relapse.
A stem cell transplant is often considered the only potentially curative treatment for people with higher-risk AML, but not everyone will be eligible for the procedure. However, certain treatment combinations can help many people achieve complete remission and remain in remission for long periods of time.
The right treatment plan depends on the type of AML, the genetic changes driving the disease, a person’s age and overall health, and whether the leukemia is newly diagnosed or has returned after treatment.
A person’s multidisciplinary care team may include hematologists, oncologists, nurses, primary care providers, dietitians, social workers, and supportive care specialists. Together, they develop a tailored treatment plan that takes the person’s needs and preferences into account.
Phases of acute myeloid leukemia treatment
AML treatment usually involves an induction phase that aims to bring the disease into remission, followed by a consolidation phase to destroy any leukemia cells that may remain and reduce the risk of the disease returning. Some people may also receive a third phase, known as maintenance therapy, to help keep the leukemia from returning for as long as possible.
Throughout all treatment phases, supportive therapies can help manage AML symptoms, treatment side effects, and other complications.
Induction
Induction therapy aims to kill as many leukemia cells as possible. It often involves chemotherapy, sometimes combined with a targeted therapy. The intensity of treatment depends on factors such as a person’s age, overall health, and the characteristics of the AML. Some people require more than one round of induction therapy before reaching remission.
Consolidation
Once remission is achieved, consolidation treatment aims to kill any leukemia cells that may remain, helping maintain remission and reduce the risk of relapse. Depending on the person’s risk factors and overall health, consolidation may involve additional chemotherapy cycles, often using the same or similar drugs as induction, targeted therapy, or a stem cell transplant.
Maintenance
Finally, if someone has genetic or other disease features that suggest an intermediate or higher risk of relapse, doctors may recommend maintenance therapy. This involves long-term treatment, often with lower-intensity chemotherapy or targeted therapy, to help prevent relapse and control any leukemia cells that may remain.
Stem cell transplant
A stem cell transplant for AML is most often performed during consolidation therapy and may offer some people a chance for long-term remission or a cure.
The procedure aims to kill as many cancerous cells as possible in the bone marrow — the spongy tissue inside some bones where blood cells are made — and infuse healthy blood stem cells, usually from another person, that can travel to the bone marrow and make new blood cells.
A stem cell transplant involves three main steps:
- Conditioning: Chemotherapy, often given at high doses and with or without radiation therapy, is used to destroy leukemia cells and prepare the bone marrow for transplant.
- Transplant: Healthy stem cells are infused into the bloodstream.
- Recovery, or engraftment: The transplanted cells usually begin producing new blood cells after about two to three weeks, although this can take longer. While a new immune system is forming, the patient is closely monitored and receives medicines to prevent and treat infections. They may also be given blood transfusions and supportive care.
Doctors may recommend a stem cell transplant for people with high-risk disease or AML that has relapsed or has not responded well to treatment. But because the procedure is intensive and carries significant risks, candidates must be healthy enough to tolerate the conditioning regimen and recovery.
Chemotherapy
Chemotherapy for leukemia, which involves using anticancer medications to destroy cancerous cells, remains the backbone of treatment for most people with AML. It may be used during induction, consolidation, and maintenance therapy, as well as before a stem cell transplant.
Many people receive intensive chemotherapy, which aims to induce remission and reduce the risk of relapse. However, some people who cannot tolerate intensive treatment may receive lower-intensity regimens that help control the disease while generally causing fewer and less severe side effects.
Several chemotherapy drugs can be used for treating AML, but people most commonly receive a combination of:
- Cytarabine: a medication that stops cells from making DNA, preventing leukemia cells from growing and multiplying.
- Anthracyclines: a class of antibiotics that damage the DNA of cancer cells, causing them to die. Examples include daunorubicin, doxorubicin, and epirubicin.
Targeted therapy
Targeted therapy for AML targets specific proteins or pathways linked to genetic changes that help leukemia cells grow and survive. These medications may be used alone or together with chemotherapy, depending on the person’s disease characteristics.
Available targeted therapies for AML include:
- FLT3 inhibitors, which are used in people with FLT3 mutations that promote leukemia cell growth. Examples include midostaurin (sold as Rydapt and generics), Vanflyta (quizartinib), and Xospata (gilteritinib).
- IDH inhibitors, which are used for people with mutations in the IDH1 or IDH2 genes that interfere with normal blood cell development. Approved IDH inhibitors include Idhifa (enasidenib), Rezlidhia (olutasidenib), and Tibsovo (ivosidenib).
- Daurismo (glasdegib), a Hedgehog pathway inhibitor that blocks signaling involved in leukemia cell survival and, in preclinical studies, made leukemic stem cells more susceptible to chemotherapy.
- Venetoclax (sold as Venclexta and generics), which targets a protein that leukemia cells overproduce to stay alive, helping restore natural cell death mechanisms.
- Revuforj (revumenib), which is used for relapsed or refractory leukemia with a KMT2A translocation or for relapsed or refractory AML with a susceptible NPM1 mutation when no satisfactory alternative treatment options are available.
- Mylotarg (gemtuzumab ozogamicin), an antibody-drug conjugate that targets the CD33 protein on the surface of most AML cells and delivers a chemotherapy payload that enters and kills the cells.
Because targeted therapies are used based on the genetic changes present in the leukemia cells, molecular testing is an important part of treatment planning.
Radiotherapy
Radiotherapy, also called radiation therapy, involves using beams of radiation to target cancerous cells, damaging their DNA and helping control their growth. This treatment is not routinely used for AML, but it may be considered in certain situations.
Specific scenarios that might involve radiotherapy include:
- before a stem cell transplant, as part of the conditioning regimen
- to treat myeloid sarcomas, tumor-like masses of leukemia cells that form outside the bone marrow
- when leukemia has spread outside the bone marrow and blood, such as to the brain and spinal fluid or the testicles
- to treat pain in an area of bone invaded by leukemia cells if chemotherapy has not helped
Some patients may require a single session of radiotherapy, while others may require repeated treatments.
Treatment for acute promyelocytic leukemia
Acute promyelocytic leukemia (APL) is a form of AML that requires a different treatment approach from most other AML subtypes.
Treatment of APL is normally divided into the same three phases used for other AML subtypes: induction, consolidation, and, for some people, maintenance. Rather than relying primarily on intensive chemotherapy, it mainly uses differentiating agents, such as all-trans-retinoic acid and arsenic trioxide, that help immature leukemia cells develop into more mature blood cells. Some people, particularly those at higher risk of relapse, may also receive chemotherapy.
Because APL can cause life-threatening bleeding and blood-clotting problems, treatment usually begins as soon as the disease is suspected, even before the diagnosis is confirmed. With prompt treatment, APL is now a highly curable form of AML, with many people achieving long-term remission.
Supportive care and managing side effects
Throughout all phases of treatment, supportive care is an essential part of managing AML. It helps ease symptoms, reduce treatment side effects, prevent complications, and maintain quality of life throughout treatment. Palliative care is one form of supportive care that focuses on easing symptoms and side effects and improving quality of life.
Supportive care may include:
- medications to prevent nausea and vomiting related to chemotherapy drugs
- red blood cell or platelet transfusions to ease certain AML symptoms
- medications to prevent or treat infections
- leukapheresis, a process that removes excess white blood cells from the blood to lower the count temporarily while chemotherapy begins working
- medications to help prevent tumor lysis syndrome, a complication that can occur when too many cancer cells die quickly
- intravenous fluids to maintain hydration
- nutritional counseling and dietary support
Rare Cancer News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.
